Correction of multi-gene deficiency in vivo using a single 'self-cleaving' 2A peptide-based retroviral vector

Nature Biotechnology

Volumn 22, Issue 5, 2004, Pages 589-594

  Szymczak, Andrea L ^a,b   Workman, Creg J ^a   Wang, Yao ^a   Vignali, Kate M ^a   Dilioglou, Smaroula ^a   Vanin, Elio F ^a,c   Vignali, Dario A A ^a,b  

^a ST JUDE CHILDREN S RESEARCH HOSPITAL   (United States)

^b UNIVERSITY OF TENNESSEE MEDICAL CENTER   (United States)

^c CENTER FOR CELL AND GENE THERAPY   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BIOLOGICAL ORGANS; BIOMEDICAL ENGINEERING; FLUORESCENCE; GENES; PROTEINS; STOICHIOMETRY;

GENE THERAPY; MULTI-GENE DEFICIENCY; MULTICISTRONIC RETROVIRAL VECTORS;

BIOTECHNOLOGY;

CD3 ANTIGEN; CELL PROTEIN; FLUORESCENT DYE; RETROVIRUS VECTOR;

ANIMAL CELL; ANTIGEN FUNCTION; ARTICLE; BLOOD; BONE MARROW; CELL TYPE; CISTRON; CONTROLLED STUDY; FLUORESCENCE RESONANCE ENERGY TRANSFER; GENE THERAPY; GENETIC DISORDER; HUMAN; HUMAN CELL; IN VIVO STUDY; MEDICAL RESEARCH; MOUSE; MULTIGENE FAMILY; NONHUMAN; PEPTIDE ANALYSIS; PICORNAVIRUS; PRIORITY JOURNAL; RELIABILITY; SEQUENCE ANALYSIS; SPLEEN; STEM CELL; STOICHIOMETRY; T LYMPHOCYTE RECEPTOR GENE; THYMUS;

AMINO ACID SEQUENCE; ANIMALS; CELL LINE; GENETIC VECTORS; HUMANS; MICE; MICE, INBRED C57BL; MOLECULAR SEQUENCE DATA; PEPTIDES; RETROVIRIDAE; SEQUENCE HOMOLOGY, AMINO ACID;

ANIMALIA; PICORNAVIRIDAE; UNIDENTIFIED RETROVIRUS;

EID: 2342471959     PISSN: 10870156     EISSN: None     Source Type: Journal    
DOI: 10.1038/nbt957     Document Type: Article

References (29)

1. Ryan, M.D. & Flint, M. Virus-encoded proteinases of the picornavirus super-group. J. Gen. Virol. 78, 699-723 (1997).
2. Palmenberg, A.C. et al. Proteolytic processing of the cardioviral P2 region: primary 2A/2B cleavage in clone-derived precursors. Virology 190, 754-762 (1992).
3. Donnelly, M. L. et al. The 'cleavage' activities of foot-and-mouth disease virus 2A site-directed mutants and naturally occurring '2A-like' sequences. J. Gen. Virol. 82, 1027-1041 (2001).
4. Ryan, M.D., King, A.M. & Thomas, G.P. Cleavage of foot-and-mouth disease virus polyprotein is mediated by residues located within a 19 amino acid sequence. J. Gen. Virol. 72, 2727-2732 (1991).
5. Donnelly, M.L. et al. Analysis of the aphthovirus 2A/2B polyprotein 'cleavage' mechanism indicates not a proteolytic reaction, but a novel translational effect: a putative ribosomal 'skip'. J. Gen. Virol. 82, 1013-1025 (2001).
6. Klausner, R.D., Lippincott-Schwartz, J. & Bonifacino, J.S. The T cell antigen receptor: insights into organelle biology. Annu. Rev. Cell Biol. 6, 403-431 (1990).
7. Persons, D.A. et al. Use of the green fluorescent protein as a marker to identify and track genetically modified hematopoietic cells. Nat. Med. 4, 1201-1205 (1998).
8. Vanin, E.F. et al. Development of high-titer retroviral producer cell lines by using Cre-mediated recombination. J. Virol. 71, 7820-7826 (1997).
9. Julias, J.G., Hash, D. & Pathak, V.K. E-vectors: development of novel self-inactivating and self-activating retroviral vectors for safer gene therapy. J. Virol. 69, 6839-6846 (1995).
10. Liu, H.Y., Rhodes, M., Wiest, D.L. & Vignali, D.A.A. On the dynamics of TCR:CD3 complex cell surface expression and downmodulation. Immunity 13, 665-675 (2000).
11. Wang, B. et al. T lymphocyte development in the absence of CD3ε of CD3γδζε. J. Immunol. 162, 88-94 (1999).
12. Love, P.E. et al. T cell development in mice that lack the zeta chain of the T cell antigen receptor complex. Science 261, 918-921 (1993).
13. Chan, F. K. et al. Fluorescence resonance energy transfer analysis of cell surface receptor interactions and signaling using spectral variants of the green fluorescent protein. Cytometry 44, 361-368 (2001).
14. de Felipe, P., Martin, V., Cortes, M.L., Ryan, M. & Izquierdo, M. Use of the 2A sequence from foot-and-mouth disease virus in the generation of retroviral vectors for gene therapy. Gene Ther. 6, 198-208 (1999).
15. Klump, H. et al. Retroviral vector-mediated expression of HoxB4 in hematopoietic cells using a novel coexpression strategy. Gene Ther. 8, 811-817 (2001).
16. Emerman, M. & Temin, H. M. Genes with promoters in retrovirus vectors can be independently suppressed by an epigenetic mechanism. Cell 39, 449-467 (1984).
17. Wang, S., Beattie, G.M., Hayek, A. & Levine, F. Development of a VSV-G protein pseudotyped retroviral vector system expressing dominant oncogenes from a lacO-modified inducible LTR promoter. Gene 182, 145-150 (1996).
18. Borman, A.M., Le Mercier, P., Girard, M. & Kean, K.M. Comparison of picornaviral IRES-driven internal initiation of translation in cultured cells of different origins. Nucleic Acids Res. 25, 925-932 (1997).
19. Mizuguchi, H., Xu, Z., Ishii-Watabe, A., Uchida, E. & Hayakawa, T. IRES-dependent second gene expression is significantly lower than cap-dependent first gene expression in a bicistronic vector. Mol. Ther. 1, 376-382 (2000).
20. Sorrentino, B.P. Gene therapy to protect haematopoietic cells from cytotoxic cancer drugs. Nat. Rev. Cancer 2, 431-441 (2002).
21. Pizzato, M. et al. Production and characterization of a bicistronic Moloney-based retroviral vector expressing human interleukin 2 and herpes simplex virus thymidine kinase for gene therapy of cancer. Gene Ther. 5, 1003-1007 (1998).
22. Moriuchi, S. et al. Enhanced tumor cell killing in the presence of ganciclovir by herpes simplex virus type 1 vector-directed coexpression of human tumor necrosis factor-alpha and herpes simplex virus thymidine kinase. Cancer Res. 58, 5731-5737 (1998).
23. Kessels, H.W., Wolkers, M.C., van, D.B., van der Valk, M.A. & Schumacher, T.N. Immunotherapy through TCR gene transfer. Nat. Immunol. 2, 957-961 (2001).
24. Zitvogel, L. et al. Construction and characterization of retroviral vectors expressing biologically active human interleukin-12. Hum. Gene Ther. 5, 1493-1506 (1994).
25. Vignali, D.A.A. & Vignali, K.M. Profound enhancement of T cell activation mediated by the interaction between the T cell receptor and the D3 domain of CD4. J. Immunol. 162, 1431-1439 (1999).
26. Markowitz, D., Goff, S. & Bank, A. A safe packaging line for gene transfer: separating viral genes on two different plasmids. J. Virol. 62, 1120-1124 (1988).
27. Persons, D.A. et al. Retroviral-mediated transfer of the green fluorescent protein gene into murine hematopoietic cells facilitates scoring and selection of transduced progenitors in vitro and identification of genetically modified cells in vivo. Blood 90, 1777-1786 (1997).
28. Persons, D.A., Mehaffey, M.G., Kaleko, M., Nienhuis, A.W. & Vanin, E.F. An improved method for generating retroviral producer clones for vectors lacking a selectable marker gene. Blood Cells Mol. Dis. 24, 167-182 (1998).
29. Carson, R.T., Vignali, K.M., Woodland, D.L. & Vignali, D.A. T cell receptor recognition of MHC class II-bound peptide flanking residues enhances immunogenicity and results in altered TCR V region usage. Immunity 7, 387-399 (1997).